Resources

Landscape on Analysis on Declining Investment in Rare Disease

Inflation Reduction Act Orphan Drug Exclusion –42 U.S. Code § 1320f–1(e)(3)(a)

CRS Report: Implementation of the Medicare Drug Price Negotiation Program: Centers for Medicare and Medicaid Guidance and Legal Considerations

CMS: Memorandum, Implementation of Sections 1191 – 1198 of the Social Security Act for Initial Price Applicability Year 2026, and Solicitation of Comments

CMS: Revised Guidance, Implementation of Sections 1191 – 1198 of the Social Security Act for Initial Price Applicability Year 2026

CMS: Negotiation Guidance Published Comments

FDA: Rare Diseases – Frequently Asked Questions

FDA Orphan Designations – Worldwide Orphan Medicinal Designations Workshop

FDA: Orphan Drug Act – Relevant Excerpts

The New England Council: Support of S. 3131 and H.R. 5539

Biotechnology Industry Organization: Comments on Medicare Drug Price Negotiation Program Initial Guidance

Biotechnology Industry Organization: New CMS guidance a ‘missed opportunity’ for future medical innovation, patients

EveryLife: Comments on Medicare Drug Price Negotiation Program Initial Guidance

National Health Council: Comments on Medicare Drug Price Negotiation Program Initial Guidance

National Organization for Rare Disorders: Comments on Medicare Drug Price Negotiation Program Initial Guidance

National Organization for Rare Diseases: Final Inflation Reduction Act Guidance Risks Stifling Innovation for Rare Disease Community

National Organization for Rare Disorders: Letter to CMS from Rare Disease Patient Organizations – Medicare Drug Price Negotiation Program Guidance

AstraZeneca Complaint

Note: This material is included for informational purposes only. The Task Force is not a party to or involved with this or any other Medicare Drug Price Negotiation litigation.

Government Response to the Complaint

University of Chicago: The Potentially Larger Than Predicted Impact of the IRA on Small Molecule R&D and Patient Health

Biotechnology Industry Organization: Understanding the IRA’s real-world impacts starts with understanding the innovation ecosystem

Biotechnology Industry Organization: New BIO Poll Reveals Americans Strongly Support the Orphan Cures Act | BIO

Real Clear Health: A Small Fix to the IRA Will Give Hope to Patients With Rare Diseases

The Hill: The Inflation Reduction Act adds new barriers to curing rare disease

BioCentury: IRA’s unintended consequences include harm to rare disease patients

Biotechnology Industry Organization: The Outlook for Orphan Drugs

Council for Affordable Health Coverage: How the Inflation Reduction Act is Impacting Rare Disease Patients

First Opinion in STAT News: The IRA needs changes to better support patients with rare diseases

Health Affairs: How The IRA Could Delay Pharmaceutical Launches, Reduce Indications, And Chill Evidence Generation

Health Capital Group: Rare Disease Companies in the Public Markets: Challenging Performance Against a Backdrop of Policy Uncertainty

Incubate: Life Sciences Investment Tracker

Pharmaceutical Research and Manufacturers of America: Reduction Act stifles innovation for small molecule medicines

Pharmaceutical Technology: Rare disease drug development needs regulatory incentives to grow in 2024

Rare Access Action Project: Inflation Reduction Act to Harm Rare Disease Innovation

Tax Foundation: Inflation Reduction Act’s Price Controls Are Deterring New Drug Development

Tufts University: Follow-On Indications for Orphan Drugs Related to the Inflation Reduction Act

University of Chicago: The Potentially Larger Than Predicted Impact of the IRA on Small Molecule R&D and Patient Health

VitalTransformation: IRA’s Impact on the US Biopharma Ecosystem